Endocrine Function During Deferasirox Therapy

• ClinicalTrials.gov Identifier: NCT04515680
• Recruitment Status: Completed
• First Posted: August 17, 2020
• Last Update Posted: August 17, 2020
• Sponsor: University of Campania "Luigi Vanvitelli"
• Information provided by (Responsible Party): Silverio Perrotta, University of Campania "Luigi Vanvitelli"

Study Description

Brief Summary:

A national survey on the prevalence and natural history of endocrine complications in thalassemia transfusion--dependent patients treated with deferasirox was designed, in order to assess a larger population during a longer follow up and improve the quality of previous investigations.

Condition or disease
Iron Overload; Transfusion-dependent Thalassemia; Endocrine; Complications

Study Design

• Study Type: Observational
• Actual Enrollment: 426 participants
• Observational Model: Cohort
• Time Perspective: Other
• Official Title: National Survey on Endocrine Function and Bone Metabolism in Transfusion--dependent Patients Treated With Deferasirox
• Actual Study Start Date: November 2, 2016
• Actual Primary Completion Date: January 30, 2019
• Actual Study Completion Date: January 30, 2019

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :

1. Change from baseline number of endocrine disorders at study completion [ Time Frame: baseline through study completion, a minimum of 5 years ]
   Absolute change in number of patients diagnosed with any endocrine disorder at the baseline and at the study completion

Eligibility Criteria

• Ages Eligible for Study: 2 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Probability Sample

Study Population

All consecutive patients visited at the participating sites since September 2009 were recruited into the cohort provided the inclusion criteria were met: affected by TDT and on deferasirox monotherapy during the study period.

Criteria

Inclusion Criteria:

• Adult and pediatric patients with transfusion---dependent thalassemia;
• Chelation with deferasirox as assigned chelation therapy;
• Available medical history including relevant clinical data (age at start of transfusion regimen, age at start of chelation therapy, prior chelation therapy, concomitant diseases and concomitant treatments, including hormonal replacement treatments if appropriate) and laboratory data (e.g TSH, FT3 and FT4, fasting serum glucose, OGTT serum glucose, bone mineral density z---score, T---score, g/cm2, PTH, FSH, LH, testosterone and estradiol, serum ferritin, liver function tests,renal function tests, MRI T2* value) at baseline and at the end of study

Exclusion Criteria:

• Non transfusion- dependent patients;
• Other chelation therapy than deferasirox or combination with other chelators during the observation;
• Absence of complete medical history as above specified

Contacts and Locations

Locations

Italy

Università degli Studi della Campania Luigi Vanvitelli

Naples, Italy, 80138

Sponsors and Collaborators

University of Campania "Luigi Vanvitelli"

More Information

• Responsible Party: Silverio Perrotta, Prof., University of Campania "Luigi Vanvitelli"
• ClinicalTrials.gov Identifier: NCT04515680
• Other Study ID Numbers: CICL670AIT16T
• First Posted: August 17, 2020
• Last Update Posted: August 17, 2020
• Last Verified: August 2020

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Thalassemia; Iron Overload; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Iron Metabolism Disorders; Metabolic Diseases